Translational Research | ALS Therapy Development Institute

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Housed within the ALS Therapy Development Institute's lab in Watertown, Massachusetts, the Augie's Quest Translational Research Center aims to better understand the mechanisms of how amyotrophic lateral sclerosis (ALS) affects the body and use the knowledge to develop new, more effective drug development.

Housed within the ALS Therapy Development Institute's lab in Watertown, Massachusetts, the Augie's Quest Translational Research Center aims to better understand the mechanisms of how ALS affects the body and use the knowledge to develop new, more effective drug development.

Naming of Augie's Quest Translational Research Center

Thanks to the tremendous leadership of Augie and Lynne Nieto, The Augie's Quest Translational Research Center honors the work, innovation and steadfast support of the entire Augie's Quest to Cure ALS organization and its many generous and committed donors, especially across the fitness and greater business community.

Steve Perrin, Ph.D., ALS TDI Vice Chairman

“It's really incredible. For 15 years, Augie's Quest has been dedicated to raising the critical funds and awareness needed to help fast-track ALS TDI's cutting-edge ALS research– specifically providing ALS TDI with more than $150 million to advance its efforts to end ALS.”

- Steve Perrin, Ph.D., ALS TDI Vice Chairman

The Role of Translational Research

"I have had the opportunity to interact with the ALS advocacy community recently, and I think it is fair to say, they are frustrated; frustrated with the lack of progress in that refractory and terrible disease. And when I look at that problem, I see a critical issue—perhaps THE critical issue—a lack of basic biologic understanding of the disease."

- Norman E. "Ned" Sharpless, MD
Acting Commissioner of the FDA

Filling A Key Knowledge Gap

Researchers still do not fully understand the mechanisms responsible for the onset and progression of ALS. This knowledge gap presents a significant challenge for ALS drug discovery. Translational research conducted at ALS TDI is focused on this critical area with the goal of rapidly and efficiently translating new information into tangible drug discovery methods and high quality drug candidates.

By learning about ALS from people with ALS, we hope to bridge the existing knowledge gap and learn more about the molecular and cellular dysfunction involved in ALS.

Translational Research at ALS TDI

Translational research is an integral part of ALS TDI's comprehensive approach to end ALS.

This graphic provides an overview of how translational research facilitates drug discovery at ALS TDI. Here are a few key take-aways:

Our translational research efforts are coordinated through the ALS Research Collaborative (ARC), utilizing biological samples and data from people with ALS to bridge the existing ALS knowledge gap; teaching us about ALS disease processes.
This better understanding about ALS disease processes informs our drug discovery efforts by revealing therapeutic targets.
Additionally, by learning directly from people with ALS, and measuring how the ALS symptoms progress, translational research efforts help us develop better clinical outcome measures.
The ultimate goals are to advance more promising treatment to clinical development and make ALS clinical trials faster and more efficient.

Learn More About Research At ALS TDI

Become a Part of Translational Research

ALS TDI's translational research effort was initiated and is carried on in tight integration with its ALS Research Collaborative (ARC). ARC is an evolution of ALS TDI's Precision Medicine Program, the most comprehensive and longest-running translational research study in ALS. Through ARC, researchers at ALS TDI partner with people with ALS around the world to share data that feeds into translational research. This integration serves ALS TDI's mission to discover and develop effective treatments for ALS.

Participate in the ARC Study

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